When my one-month-old daughter was diagnosed with biliary atresia — a rare disease that for most of human history was 100% fatal — I entered a world where hope and terror coexist.

Thanks to pioneers who pushed the envelope when everyone else had given up, at thirteen months old she underwent a liver transplant in Pittsburgh, and today she is alive and thriving in college.

So I understand intimately why families facing devastating illnesses want access to innovative therapies. But innovation only works if the science ecosystem can differentiate true innovation from failure.

A recent Wall Street Journal column argues that the U.S. Food and Drug Administration (FDA) is standing in the way of hope for rare disease patients. It frames the debate as a simple moral choice: let patients try promising therapies or deny them a chance at survival.

That framing is dangerously incomplete.

Most reasonable people agree that rare disease patients should have the right to try innovative therapies, provided the data are strong and the risks understood. But today, that right can be hijacked by investors with a financial stake in characterizing treatments as safe and effective on the basis of partial data. When investors sell false hope, patients lose the impartial referee they need.

Consider the gene therapy AMT-130, currently under development to treat the........

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